Modality-agnostic lab has already identified 8 novel treatments for previously untreatable rare diseases working directly with 30 families across four continents — rooted in a single conviction: Time Is Life.

 RareLabs, a division of AlphaRose Therapeutics, a modality-agnostic precision drug discovery lab, today announced its public launch and the appointment of Dr. Rob Freishtat as Chief Executive Officer. Dr. Freishtat, who continues as Chief Medical Officer of AlphaRose Therapeutics, will lead RareLabs as it comes out of stealth and continues its work as a first-of-its-kind AI- and robotics-enabled platform that designs and validates genetic medicines — antisense oligonucleotides (ASOs), siRNAs, gene therapies, and repurposed-drug regimens, with timelines measured in weeks, not years.

“For patients and families facing a rare genetic diagnosis, every month of delay is potentially a month of lost function — often, lost irreversibly. RareLabs exists to collapse the drug discovery timeline. Time is life for patients facing the impacts of rare conditions.”— Dr. Rob Freishtat, CEO, RareLabs; CMO, AlphaRose

What RareLabs Does

RareLabs is a modality-agnostic precision drug discovery lab built to rapidly create treatments for genetic diseases. Its integrated AI- and robotics-enabled system designs, screens, and validates candidate therapies across four modalities, all under one roof simultaneously:

• Antisense oligonucleotides (ASOs) for splicing and gene-silencing interventions
• Small interfering RNAs (siRNAs) for targeted mRNA knockdown
• Gene therapies including AAV-based and non-viral delivery approaches
• Repurposed-drug screens mining approved compounds for rare-disease applications

Because the platform is modality-agnostic, RareLabs matches each patient and disease to the right therapeutic approach, rather than forcing a one-size-fits-all, therefore accelerating therapeutic options for patients.

Proven Traction for Previously Untreated Diseases

Since its founding, RareLabs has been led in the lab by Dr. Rodney Bowling Jr., whose prior discovery of more than 20 therapeutics and involvement in dozens of clinical trials laid the foundation for the platform. Under his direction, RareLabs has:

• Discovered eight novel potential treatments for rare diseases that previously had no therapeutic options
• Partnered with 30 disease communities and families spanning more than four continents
• Operated across the full discovery-to-validation arc, from target selection through in vitro efficacy demonstration

Patient Spotlight: Saving Sasha

RareLabs recently designed a custom ASO therapy for Sasha, a patient with an ultra-rare mutation in SLC6A1, a gene implicated in severe neurodevelopmental and epileptic conditions. The custom ASO demonstrated efficacy in patient-derived cell lines, a critical milestone on the path from n=1 discovery to compassionate-use treatment.

“Sasha’s story is why we built RareLabs. When a person is facing an ultra-rare mutation, there is no pipeline waiting for them. We build the pipeline, fast, rigorous and tailored to that single person. Cell-line efficacy is how you turn ‘impossible’ into ‘let’s keep going.'”— Casey McPherson, CEO, AlphaRose Therapeutics

Global Expansion: Middle East Partnerships

RareLabs leadership recently returned from the Middle East, where active discussions are underway to establish RareLabs partnerships across the region, broadening access to genetic-medicine discovery for families in a region that has historically been underserved by Western rare-disease infrastructure.

Leadership: Dr. Rob Freishtat, Chief Executive Officer

Dr. Rob Freishtat is a physician-scientist and translational medicine leader with more than two decades of experience at the intersection of pediatric medicine, genomics, and therapeutics development. As CMO of AlphaRose and now CEO of RareLabs, he brings deep clinical insight and operational rigor to the company’s mission of delivering rare-disease treatments at the pace patients need.

Under his leadership, RareLabs will continue to scale its AI and robotics platform, expand partnerships with patients, foundations and biotech collaborators worldwide, and advance its portfolio of candidate therapies toward the clinic.